Safety and Proof-of-Concept (POC) Study With AMT-130 in Adults With Early Manifest Huntington's Disease
Public ClinicalTrials.gov record NCT04120493. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 1/2, Randomized, Double-Blind, Sham Control and Open-Label Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Therapy (AMT-130) in Early Manifest Huntington's Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This is the first study of AMT-130 in patients with early manifest HD and is designed to establish safety and proof-of-concept (PoC). CT-AMT-130-01 is a Phase 1/2, multicenter, first-in-human (FIH) study. The first three cohorts of the study have completed enrollment, including the randomized, double-blind, sham-controlled cohorts. Cohort 4 is open-label. Cohort 4 participants will receive high dose AMT-130.
Study identification
- NCT ID
- NCT04120493
- Recruitment status
- Active, not recruiting
- Study type
- Interventional
- Phase
- Phase 1, Phase 2
- Enrollment
- 43 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 25 Years to 65 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Sep 6, 2019 (Actual)
- Primary completion
- Jun 2029 (Estimated)
- Completion
- Dec 2029 (Estimated)
- Last update posted
- Oct 21, 2025
2019 – 2029
United States locations
- U.S. sites
- 12
- U.S. states
- 12
- U.S. cities
- 12
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294-0111 | — |
| University of Arizona (Surgical Site Only) | Tucson | Arizona | 85724 | — |
| University of California, San Francisco | San Francisco | California | 94158 | — |
| CenExel Rocky Mountain Clinical Research | Englewood | Colorado | 80113 | — |
| Rush University Medical Center | Chicago | Illinois | 60612 | — |
| Johns Hopkins University | Baltimore | Maryland | 21287 | — |
| University of Michigan Department of Neurology | Ann Arbor | Michigan | 48105 | — |
| Ohio State University | Columbus | Ohio | 43210 | — |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | — |
| The University of Texas | Houston | Texas | 77030 | — |
| Virginia Commonwealth University VCU School of Medicine, Department of Neurology | Richmond | Virginia | 23298 | — |
| University of Washington Medical Center | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04120493, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Oct 21, 2025 · Synced Oct 18, 2026
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Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT04120493 live on ClinicalTrials.gov.